Spark Unveils Vision of Having 10 Clinical-Stage Gene Therapy Programs by 2018, Including One Commercial and Two in Pivotal Trials

PHILADELPHIA, Jan. 11, 2016 (GLOBE NEWSWIRE) -- Spark Therapeutics Inc. (ONCE) today outlined a comprehensive three-year vision for expanding the company's portfolio through 2018 and launching its first commercial product in 2017, during a presentation this afternoon at the J.P. Morgan 34th Annual Healthcare Conference in San Francisco.

Spark Co-founder and Chief Executive Officer, Jeffrey D. Marrazzo, outlined plans for advancing 10 total programs across three therapeutic franchises by 2018, including one commercially approved therapy, two product candidates in pivotal-stage trials and at least seven additional programs in clinical proof-of-concept trials. The company's 2018 vision for its portfolio is fully funded with existing financial resources, Marrazzo said.

"The overwhelmingly positive data from our pivotal Phase 3 program for RPE65-mediated blindness, together with the multi-year durability data presented from the same program, provide strong validation of the Spark platform for AAV-based gene therapy that we are deploying across a large and growing pipeline of product candidates," Marrazzo said. "Our results reflect the power of a true platform that combines proven capabilities across vector selection, design and manufacture, a history of collaborating with regulators to optimize clinical development and develop novel clinical endpoints, and our position at the forefront of shaping a patient-centric, commercial model for gene therapies. We are now leveraging this platform through internal innovation and commercialization, partnering and external collaboration to transform the treatment of a wide range of severe genetic diseases in three target tissues – the eye, the liver and the central nervous system (CNS)."

Among the programs unveiled for the first time were two new indications, including Leber hereditary optic neuropathy (LHON), an additional inherited retinal dystrophy (IRD) program, which affects over 7,500 patients, with an even greater number of patients at risk for losing their sight in the United States and the five major European markets (EU5). Within the Company's growing CNS portfolio, Spark unveiled its program targeting Huntington's disease (HD), a hereditary genetic disorder that affects over 60,000 patients in the United States and the EU5.

The Company also outlined multiple data readouts and regulatory milestones that it expects over the next 18 months across five separate product candidates, including:

  • SPK-RPE65: A Biologics License Application (BLA) filing in the second half of 2016 and a Marketing Authorization Application (MAA) filing in early 2017, along with the readout of one-year efficacy data for the Phase 3 crossover subjects (n = 9), two- and four-year durability data from the Phase 3 intervention group (n = 20) and a comparable cohort of subjects (n = 8) from an earlier Phase 1 trial, respectively;
  • SPK-CHM: Initial safety and efficacy data in the second half of 2016 from the Phase 1/2 trial for choroideremia;
  • SPK-FIX: Initial efficacy data in mid-2016 for the Phase 1/2 trial for hemophilia B;
  • SPK-FVIII: An Investigational New Drug (IND) filing in the second half of 2016 and initial efficacy data in hemophilia A in the first half of 2017; and
  • SPK-TPP1: An IND filing in the second half of 2016 and initial efficacy data for TPP1 deficiency, a form of Batten disease, in the first half of 2017.

Additionally, Spark announced four recent developments relating to its lead program, SPK-RPE65:

  • Further analyses of the visual acuity data from the Phase 3 trial yielded statistically significant results: (i) using the Lange scale, intervention group subjects improved an average of 9 letters versus 1.6 for the control subjects (p = 0.047); and (ii) in a sub-group analysis that removes subjects that developed cataracts (n = 3), as recommended by the Company's Data Safety Monitoring Board, intervention group subjects improved an average of 10.6 letters versus 1.6 for controls (p = 0.007);
  • The company received positive scientific advice from the European Medicines Agency (EMA) regarding the requirements for its MAA filing including the feasibility of pursuing an approach to the ultimate label that is similar to the approach being pursued with the Food and Drug Administration (FDA), combining a core clinical manifestation of the disease with a genetic mutation, RPE65;
  • The company converted the license from the University of Pennsylvania, the University of Florida and Cornell University with claims covering the method of treating RPE65-mediated diseases from co-exclusive to exclusive; and
  • SPK-RPE65 received its non-proprietary product name, voretigene neparvovec.

Spark closed 2015 with more than $290 million in cash and equivalents, following the completion of a follow-on offering in December 2015. This figure does not reflect a $15 million milestone payment from Pfizer that was earned in December 2015.

About Spark Therapeutics
Spark is a gene therapy leader seeking to transform the lives of patients with debilitating genetic diseases by developing one-time, life-altering treatments. Spark's initial focus is on treating rare diseases where no, or only palliative, therapies exist. Spark's most advanced product candidate, SPK-RPE65, which has received both breakthrough therapy and orphan product designation, recently reported positive top-line results from a pivotal Phase 3 clinical trial for the treatment of rare blinding conditions. Spark's validated gene therapy platform is being applied to a range of clinical and preclinical programs addressing serious genetic diseases, including inherited retinal dystrophies, hematologic disorders and neurodegenerative diseases. Spark builds on two decades of research, development and manufacturing at The Children's Hospital of Philadelphia, including human trials conducted across diverse therapeutic areas and routes of administration. To learn more, please visit

Cautionary Note on Forward-looking Statements
This release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the company's lead product candidate, SPK-RPE65 and its other pre-clinical and clinical product candidates. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that: (i) the data from our Phase 3 clinical trial of SPK-RPE65 may not support a label for the treatment of RPE65-mediated IRDs other than Leber congenital amaurosis (LCA); (ii) the improvements in functional vision demonstrated by SPK- RPE65 in our clinical trials may not be sustained over extended periods of time; (iii) we could experience delays in submitting our regulatory filings, including our Biologics Licensing Application with FDA and our marketing authorization application with the European Medicines Agency for SPK-RPE65 and, once submitted, such regulatory filings may not be approved; (iv) our product candidate, SPK-CHM, will not demonstrate sufficient clinical data to warrant continued development; (v) our collaboration with Pfizer will not be successful; and (vi) any one or more of our product candidates in preclinical or clinical development will not successfully be developed and commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in our Annual Report on Form 10-K, our Quarterly Reports on Form 10-Q and other filings we make with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Spark undertakes no duty to update this information unless required by law.



Investor Relations
Spark Therapeutics, Inc.
Stephen W. Webster
Chief Financial Officer
(855) SPARKTX (1-855-772-7589)

Ten Bridge Communications
Dan Quinn
(781) 475-7974
[email protected]